A First-in-Human, Open-Label, Dose-Escalation Study to Evaluate the Safety and Tolerability of Gene Therapy with TTX-381 for the Ocular Manifestations Associated with Neuronal Ceroid Lipofuscinosis Type 2 (CLN2) Disease

PHASE1/PHASE2RecruitingINTERVENTIONAL
Enrollment

16

Participants

Timeline

Start Date

May 17, 2023

Primary Completion Date

July 30, 2026

Study Completion Date

July 30, 2030

Conditions
Neuronal Ceroid Lipofuscinosis Type 2
Interventions
GENETIC

TTX-381

One time subretinal dose in study eye

Trial Locations (1)

Wc1N 3JH

RECRUITING

Greater Ormond Street Hospital, London

All Listed Sponsors
lead

Tern Therapeutics, LLC

INDUSTRY